Stem Cell Clinic

Antiviral T cells protected, viable as off-the-shelf treatment for painful complication stem cells – Microbioz India

Infusion of T cells targeting BKV resulted in rapid responses, with 67.7% of patients seeing a complete or partial improvement in symptoms after 14 days. This increased to 81.6% of patients after 28 days post-infusion. No cases of grade 3 or grade 4 graft versus host disease (GVHD) or other infusion-related toxicities occurred.

Addressing a painful complication caused by BKV

BKV-associated hemorrhagic cystitis is an incredibly painful condition that causes significant morbidity in patients and can lead to worse cancer outcomes in the long term. Unfortunately, there are no effective treatments available. We were driven to develop this therapy to provide a better option for these patients, and this has emerged as a safe and effective therapy for patients at MD Anderson based on these results.

Katy Rezvani, M.D., Ph.D., Corresponding Author, Professor of Stem Cell Transplantation and Cellular Therapy

BKV is a human polyomavirus acquired by most people in early youth, Rezvani explained. Held in check by the immune system, BKV typically stays dormant in the cells lining the urinary tract, including the kidney, liver, bladder and ureters.

Allogeneic stem cell transplants, where stem cells come from a matched donor, require patients to be given therapies to suppress the immune system and protect against rejection. However, this can also trigger the BKV to start reproducing and cause severe cystitis, which could lead to hospitalization for days or months. An antiviral called cidofovir was used as a treatment but is associated with significant toxicities.

Recognizing that these viral infections can profoundly impact patient healing, Rezvani and her research team directed the development of antiviral T-cell therapies with the support of the MDS and AML Moon Shot, part of the institutions Moon Shots Program, a collaborative effort to quickly develop scientific discoveries into meaningful clinical improvements that save patients lives.

Starting with blood samples taken from healthy donors, the study team can isolate T cells and grow them to specifically recognize and target an assortment of antigens located on the BKV. These cells are expanded in the clinical good manufacturing practice (GMP) lab, under the direction of Elizabeth J. Shpall, M.D., professor of Stem Cell Transplantation and Cellular Therapy. From every donor, the team can fabricate anywhere from 20 to 50 doses of antiviral T cells, which can be stored until needed, Rezvani said.

In an earlier trial published in the New England Journal of Medicine, these researchers showed that BKV-specific T cells may effectively treat disease with the JC virus, a genetically similar polyomavirus which causes progressive multifocal leukoencephalopathy (PML), a rare and frequently fatal brain infection.

BKV-specific T cells get positive results in trial The current trial enrolled a total of 59 MD Anderson patients experiencing BKV-HC after an allogeneic stem cell transplant. Women accounted for 40.7% of participants and men for 59.3%.

Patients received a single infusion of partially human leukocyte antigen (HLA)-matched T cells, with the option to get additional infusions every 2 weeks, if necessary.

Following infusion, the researchers did not observe any side effects which were probably attributed to the antiviral T cells. Several patients developed delayed cases of low-grade GVHD in the weeks and months after therapy, which were well within the expected rates of GVHD for these patients early after allogeneic stem cell transplant, Rezvani explained.

The median time for patients to have a partial response was 14 days, and the median time to complete response was 21 days. The estimated probability of achieving a complete response was almost 70% by day 45, indicating continuous activity of the infused cells. Responses were durable and no patients saw their symptoms return after a formerly achieved response.

After analyzing participants on the trial, the researchers determined that expansion of the infused T was positively correlated with individual responses.

Were extremely encouraged by the safety of the treatment and the fast answers we have seen in the vast majority of patients, Rezvani said. Because this method is so safe, we have been able to provide this therapy as an outpatient procedure as soon as patients start developing symptoms. This has been life-changing for the patients weve been able to treat thus far.

Ultimately, the researchers aim to validate these findings at a multi-institutional research and bring this treatment option to many more patients in need.

A complete list of cooperating authors and their disclosures are available with the full paper here. In addition to the Moon Shots Program, the study was supported by the National Institutes of Health (R01CA211044-05, CA016672).

Source:

Journal reference:

Olson, Z.,et al.(2021) Third-Party BK Virus-Specific Cytotoxic T Lymphocyte Therapy for Hemorrhagic Cystitis Following Allotransplantation.Journal of Clinical Oncology.doi.org/10.1200/JCO.20.02608.

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Antiviral T cells protected, viable as off-the-shelf treatment for painful complication stem cells - Microbioz India

Investigating CAR T-Cell Therapy for Use in Different Disease Types – Targeted Oncology

Michael Bishop, MD, a professor of medicine and director of Hematopoietic Stem Cell Transplantation Program at The University of Chicago Medicine, discusses the investigations of and responses to chimeric antigen receptor (CAR) T-cell therapy in different disease types.

One of the settings furthest along in exploring the use of CAR T cells is non-Hodgkin lymphoma, with about 40% to 50% patients are achieving sustainable complete responses with CAR T-cell therapy. There are still significant areas that can be improved upon for this patient population, according to Bishop.

Another disease type where there is potential opportunity to use CAR T cells is acute lymphoblastic leukemia. Bishop says there has been success for children in this setting, and now there is study for adults that will be reporting out soon called the ZUMA-3 trial (NCT02614066), which investigated brexucabtagene autoleucel (KTE-X19). Other studies are looking at the use of allogeneic CAR T-cell therapy in patients with acute lymphoblastic leukemia.

There are also trials in multiple myeloma looking at CAR T-cell therapy. Bishop feels the results for this patient population appear to be very exciting, with higher and more sustained complete response rates, and there is an opportunity to further improve upon those results.

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Investigating CAR T-Cell Therapy for Use in Different Disease Types - Targeted Oncology

Hope, hype and exploitation the wild history of stem cell science – ABC News

Stem cells and regenerative medicine have been heralded since the mid-20th Century as the best hope for curing countless physical ailments.

Decades later, there have been perhaps as many scandals in this field of medicine, as there have been genuine advances in research and application; and the full potential of stem cells is yet to be realised.

Physician-scientist Professor John Rasko de-mystifies the science by sharing stories of some of the field's most influential and infamous figures.

He also explains why there's still plenty of reason to be hopeful.

Flesh Made New: The Unnatural History and Broken Promise of Stem Cells by John Rasko and Carl Power is published by Harper Collins

Professor John Rasko is the Head of Department of Cell and Molecular Therapies at Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program at the centenary Institute, University of Sydney

John is a featured guest of Griffith University's Integrity 20

Listen to John's 2018 Boyer Lectures

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Hope, hype and exploitation the wild history of stem cell science - ABC News

Should scientists be allowed to grow human embryos in a dish beyond 14 days? Is it scientifically important or morally wrong? – USA TODAY

Human blastocyst, Getty Images

For more than 30years, scientists have followed a rule they imposed on themselves to avoid growing a human embryo in a lab dish for more than 14 days.

Until recently, the "14-day rule" was largely academic. Scientists couldn't grow themfor that long if they wanted to.

But in 2016, two teams of researchers reached 12days, and in 2019, another group grew monkey embryos for 19 days.

These advances have spurredsome scientists to argue in two recent papersthat the 14-day rule should bemodified or dropped. There's a lot to be learned by pushing embryos out to 28 days, they say.

The regulatory committee of theInternational Society for Stem Cell Research, which lays down guidelines for the scientificfield,has been debating the issuefor months and is expected to issue its final decision this month.

Some ethicists and scientistsare concerned that revising the rule just asit becomes technologically feasible to break it is ridiculous and morally repugnant.

"If you abandoned every rule or law that inhibits you as soon as it inhibits you, we'd live in a lawless world," said Ben Hurlbut, a historian of science at Arizona State University.

And somepeople consider human embryo researchto be unethical at any stage.

"Whether 14 days, 14 months, or anywhere in between, such 'rules'remain contrivances to justify the most unethical kinds of science and to allow for the exploitation of our own vulnerable human offspring," said Tadeusz Pacholczyk, a neuroscientist and director of education at the National Catholic Bioethics Center in Philadelphia.

A single cell is removed from a human embryo to be used in generating embryonic stem cells for scientific research.Advanced Cell Technology via AP

Countries are free to ignore rules set by the society, but scientists for decades have generally abided by them. (In the U.S., there's no national law about the 14-day rule, though some states have their own regulations.)

Some cultures and religions believe that human life begins at conception, or that the human embryo carries a special status from conception onward. Other cultures believe that life starts later in fetal development, or even at birth.

Biologists routinely grow amphibian and mammal embryos in petri dishes, but human embryos are different.

Until about 14 days after conception, the human embryo looks likean undifferentiated blob of cells, which is one of the reasons the two week timeframemade sense, several scientists said.

Robin Lovell-Badge, who sits on the International Stem Cell Society committee that's considering overturning the rule, said scientists will take any changes seriously.

"We've stuck with that rule for over 30 years," he said.

Lovell-Badgefavors extending the limit, as long as the research is scientifically justified and has public support.

Not everyone in the scientific community shares this position.

"It's been a difficult part of the guidelines to get agreement on," Lovell-Badge said. "You have very wide-ranging views."

Some scientists argue there's a lot to be learned by pushing the 14-day rule out another two weeks.

Right now the second two weeks after fertilization is considered a "black box" because so little is known about it, said Insoo Hyun, a professor of bioethics atCase Western and Harvard universities. Heco-wrote a March 5 opinion piece arguing for a careful, stepwiseextension of the 14-day rule.

"You have to really make your case for it,"Hyun said."You have to explain what you want to do and why, have a very clear picture of where the next stopping point is."

Women generally don't know they're pregnant before 28 days, so historically, there has not been tissue from aborted or miscarried fetuses available for research.

The central nervous system, heart and other organs begin to develop during this crucial two-week period. The body plan is established. Cells that will become eggs and sperm start to form. Aspects of theplacenta are set up.

In many ways,days 14 through28 are the most interesting period of human development, Lovell-Badge said. "You can do a whole lot of incredibly valuable research," in that timeframe, he said.

And it's in that window that many things can go wrong in a pregnancy, such as miscarriage or abnormalities.

Perhaps there are treatments that could be developed to fix these problems, if they are better understood,Hyunsaid, just as pregnant women now take vitamin supplements to prevent spina bifida, in which the spine doesn't developproperly.

Do you think scientists should be allowed to grow embryos in a dish beyond 14 days? Share this story.

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Developing embryos for another week "will thus illuminate this poorly understood period of our development and bring greater understanding of pregnancy loss and developmental disease,"saidMagdalenaZernicka-Goetz,theBritish scientist who developed the technique for growing human embryos for nearly two weeks.Zernicka-Goetz,author of a 2020 book on human development called "The Dance of Life,"would like to extend the 14-day rule out one week to 21 days.

"This will enable thescientiststo study a period of development that are highly susceptible to developmental failure, something that happens quite frequently in human pregnancy," she wrote in an email, stressing work should be closely regulated"to achieve these potential biomedical advances within an appropriate bi-ethical framework."

Despite their differences, most scientists seem to agreethere's no reason to push development past 28 days.

By onemonth after conception, embryonic tissue is easier to obtain and study and theorgans have formed, leaving fewer questions to answer.

"You wouldn't need to take them much beyond that point anyway," Lovell-Badgesaid.

Pacholczyk, of the Catholic Bioethics Center, said there's simply no justification for 14 days or any other time limit.

"Researchers have been feigning for a long time that the 14-day rule was somehow an ethical tenet grounded in biological facts while in reality it has been little more than a ceremonial 'line in the sand' and it should come as little surprise that they are now seeking to move that line beyond 14 days," he wrote in an email.

Even some who strongly supportscientific research are uncomfortable extending the 14-day rule.

HenryGreely, who directs the Stanford Center for Law and the Biosciences at Stanford University in California, saidthere should be a hard-stop endpointfor embryo research.

"Even though I do not personally give strong moral status to embryos, the idea of doing research on 18-day-oldhuman embryos is disturbing," said Greely, author of the new book "CRISPR People: The Science and Ethics of Editing Humans."

This sequence of images shows the development of embryos after correcting for a genetic error that would otherwise cause a type of inherited heart disease.OHSU

"I'd like to see an endpoint that had some rationale that would make it likely to stick," he said.

Growing an embryo in a lab dish instead of a woman's womb is necessarily different, Greely said, and may not represent a "real" embryo anyway.

"Does a 14-day embryo that is not implanted deep in a woman's uterus tell us anything meaningful about a 14-day embryo that is?" he asked.

Marcy Darnovsky, executive director of the Center for Genetics and Society, a nonprofit advocacy group,said efforts to overturn the 14-day rule are another example of scientific over-reach.

"There's a real problem with scientists who are jumping ahead of the public," she said.

Scientists should not be the ones who get to decide where society's moral boundaries lie,she and Hurlbut said.

"If moves are made to usurp these questions from wider society," Hurlbut said, "it's to the detriment of democracy and to the detriment of sciencecertainly in the long run, and probably in the short run."

Contact Karen Weintraub at kweintraub@usatoday.

Health and patient safety coverage at USA TODAY is made possible in part by a grant from the Masimo Foundation for Ethics, Innovation and Competition in Healthcare. The Masimo Foundation does not provide editorial input.

Published 10:13 am UTC May. 2, 2021 Updated 10:13 am UTC May. 2, 2021

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Should scientists be allowed to grow human embryos in a dish beyond 14 days? Is it scientifically important or morally wrong? - USA TODAY

Brave New World is being reinvented with synthetic embryosand the right reasons – SYFY WIRE

In his 1932 novel Brave New World, Aldous Huxley walks you into a terrifying lab where human embryos and fetuses are being grown in glass containers and genetically engineered to fit into a certain rank of society. It continues to be nightmare fuel for college students everywhere.

Now that we live in an era where science fiction is morphing into science, conceiving artificial embryos sounds like an incarnation of the bookbut couldnt be further from it. Scientists have proven it is possible to synthetically create embryos from stem cells. This is a viable and ethical alternative to studying human or animal embryos, and a new frontier in finding out more about how preimplantation embryos may mutate or fail.

Instead of trying to build a societal hierarchy from human beings born in vitro, researchers Cody Kime, Kiichiro Tomoda and their team from Kyoto University and the RIKEN Center for Biosystems Dynamics Research are using their findings in an effort to find out what can go wrong with embryos in their earliest phases and cause early pregnancy loss. They recently published a study inStem Cell Reportsandare optimistic that the glitches of nature can someday be prevented.

"As you can imagine, there is tremendous power, inevitable risk, and serious ethical responsibility, although using cultured cells we can greatly reduce animal experiments. Perhaps one of the best applications is screening genetic mutations that impede fertility and reproduction,"Kime told SYFY WIRE. "if those mutations are tolerated in our starting stem cell population, we can initiate reprogramming, and see how those mutations affect the synthetic embryo system. From there, we can get a better picture of how those genes may affect human fertility and improve on treatments."

If organoids (even the brain) can be grown for further research, so can embryoids. The team, whose in vitro synthetic embryo systems (SESs)came from mouse stem cells, was trying to successfully recreate totipotency, meaning that the cells would have everything they needed to develop into a whole organism. Totipotency does not last long in embryonic stem cells. Pluripotency, which is the ability to produce some types of necessary cells, but not all, is much easier to achieve. This is still a positive. If the stem cells are at least pluripotent, it might be possible to reprogram them to be totipotent.

There are three types of cells needed for totipotency to happen. They need to be able to generate the embryo, the placenta and the amniotic sac. This is such a fleeting state in mammals because the cells in the embryo multiply and polarize fast to turn into one of those three things.Kime and Tomoda didnt try to do everything at once. The team started by growing pluripotent mouse cells, or epiblast stem cells, responsible for only the fetus. Pluripotent pre-implantation epiblast stem cells are capable of arranging themselves into what at least looks like an embryo before it implants. If they could somehow hit the rewind button on those cells, they would revert them back to a stage before they specialized in just the fetus, the totipotent phase.

"We have seen evidence that something like totipotency may be happening in our reprogramming system, and it arises by taking a later stage embryonic stem cell and treating it with specific natural molecules and nutrients," Kime said."In a way the cell is tricked to reprogram and gain the ability to form the other embryonic lineages."

Reprogramming meant that the scientists would need to be able to tell which genes each cell turned on oroff. They used a process called RNA sequencing, which sees how much RNA is and how many sequences of that RNA is in a sample. RNA (ribonucleic acid) tells DNA how to put together different proteins. Sequencing reveals its transcriptome, or everything that makes up RNA, and allows scientists to better understand cells up close. They observed the gene expression in thousands of cells, which told themwhich cells could be potentially reprogrammed to become totipotent. Hi-res regulation of gene expression could even show what ways cells were changing.It took Kime 5 days of reprogramming attempts, but some of the past epiblast stem cells (EPISCs) finally got there.

The analysis revealed that cells resembling all three types of the early embryo were generated by our unique reprogramming system at the same time," he said. "Our analysis showed, in great detail, that our reprogrammed cells had engaged nearly all early embryo cells, while turning off the genes of the cell type they came from. The most important analysis was comparing our reprogrammed cells to real embryonic cells and finding that, across incredibly rich data, our cells were nearly identical."

The breakthrough has given Kime, Tomoda and their team a portal into what was once unthinkable. Because epiblast stem cells are easily reproduced, they can carry out studies on a much larger scale. They will also be able to explore things that would have not been considered ethical otherwise, such as getting a more in-depth look at how reprogramming happens and screening for gene mutations and other things that could cause a pregnancy to terminate itself. So while their work may be venturing into a brave new world, the intent is the total opposite of the sinister motives in Brave New World.

Whether strawberry ice cream soma will ever be a thing still remains to be seen.

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Brave New World is being reinvented with synthetic embryosand the right reasons - SYFY WIRE

Stem Cell Therapy Market Trends Evaluation 2021 By Leading Players Updates, Consumer-Demand, Key Strategies, Consumption, Industry Development, Market…

The report offers information on the variety of products and services provided in the market, covering a multitude of operations and activities throughout the global Stem Cell Therapy market value chain. The study analyzes the world Stem Cell Therapy market in terms of production, income, sectoral structure, and export and trade activities. Moreover, technological tendencies in the global Stem Cell Therapy market have been put forward. The growth rate of the market in developed countries like Mexico, Brazil, Argentina, Australia, the EU, Canada, the USA, and in developing countries like India and China is studied in detail. The most important technological advancements in the market are presented in the study.

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1. Economic and trade-related activities in the global Stem Cell Therapy market. 2. Changes in production systems over the years. 3. The future of the global Stem Cell Therapy market, trends, and challenges. 4. Productivity growth constraints. 5. The shift in consumer preferences and key factors changing the market dynamics. 6. Current and anticipated market size and CAGR in USD billion. 7. Key features and growth rate of the global Stem Cell Therapy industry worldwide. 8. Macro outlook and industry outlook. 9. Market landscape pertaining to the global Stem Cell Therapy market selected countries. 10. Trade scenarios, major export destinations, major countries of import. 11. Digital initiatives by the leading players for capacity building and boosting operational efficiency. 12. Quality, serviceability, financial, policy-related, capacity building constraints associated with the unorganized manufacturers in the market. 13. The report summarizes export opportunities in selected markets.

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By type, Stem Cell Therapy Market is segmented as:

Based on cell source, the market has been segmented into,

Adipose Tissue-Derived Mesenchymal SCs Bone Marrow-Derived Mesenchymal SCs Embryonic SCs Other Sources

By Applications, Stem Cell Therapy Market is segmented as:

Based on therapeutic application, the market has been segmented into,

Musculoskeletal Disorders Wounds & Injuries Cardiovascular Diseases Gastrointestinal Diseases Immune System Diseases Other Applications

The international trade, investment, and export promotional strategies implemented by the industrialists, traders, and investors to strengthen the global economic competitiveness are included in the report. The knowledge on these developments given in the report unlocks export and investment opportunities for the stakeholder and other participants in the industry. Quantitative and qualitative analysis to combine the results is done. This study groups the export markets for the products and services in the global Stem Cell Therapy market into three categories: Strategic, Dynamic Growth, and Long-Term Opportunity.

These export markets are ranked based on the criteria including volume ranking, growth ranking, and performance ranking. The volume ranking ranks the global export markets by dollar value, highest to lowest whereas the growth ranking is based on the averaging the Compound Annual Growth Rate (CAGR) of these markets for the years 2010-2015 and 2016-2020. The CAGR of the years 2016-2020 is included to capture the more recent developments. And, performance ranking globally is based on the exports of the main categories. The global commodity prices and the other factors influencing the corresponding categories are taken into consideration. The major driving powers of technological trends such as market, manufacturers, and legislation are studied in detail.

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1. This report covers a comprehensive market overview presenting the competitive market scenario among key players, understanding their growth opportunities, and business strategies 2. The factors contributing to the growth, market risks, growth restraining factors are analyzed in this report to help other players in making an informed decision 3. The five-year forecast information will pave the way for futuristic developments and expected market growth 4. A thorough analysis of key Stem Cell Therapy product segments, changing market dynamics will lead to complete market study 5. This research study serves as a complete guide which provides valuable insights and in-depth analysis of various industry verticals

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1. Industry Overview of Stem Cell Therapy 2. Industry Chain Analysis of Stem Cell Therapy 3. Manufacturing Technology of Stem Cell Therapy 4. Major Manufacturers Analysis of Stem Cell Therapy 5. Global Productions, Revenue and Price Analysis of Stem Cell Therapy by Regions, Creators, Types and Applications 6. Global and Foremost Regions Capacity, Production, Revenue and Growth Rate of Stem Cell Therapy 2015-2020 7. Consumption Volumes, Consumption Value, Import, Export and Trade Price Study of Stem Cell Therapy by Regions 8. Gross and Gross Margin Examination of Stem Cell Therapy 9. Marketing Traders or Distributor Examination of Stem Cell Therapy 10. Worldwide Impacts on Stem Cell Therapy Industry 11. Development Trend Analysis of Stem Cell Therapy 12. Contact information of Stem Cell Therapy 13. New Project Investment Feasibility Analysis of Stem Cell Therapy 14. Conclusion of the Global Stem Cell Therapy Industry 2020 Market Research Report Continued

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STEM NOLA Teams with Obatala Science & CellSpring to Expose Students to Careers and Medical Research at Biotech Startups – PRNewswire

NEW ORLEANS, April 30, 2021 /PRNewswire/ --STEM NOLA is proud to partner with Obatala Sciences and CellSpring to engage at least a dozen high school students from the greater New Orleans area in a series of courses that will expose teens to careers in biotechnology startup companies. This extraordinary program focuses on providing high school students with exposure-rich, hands-on opportunities to engage deeply in work in the realm of STEM.

Over the course of three Saturday sessions on May 8, 15 and 22, students will receive comprehensive lab exposure to the work at both companies involving tissue engineering, stem cell research (stem cells isolated from adult fat tissue), and preclinical evaluation of potential therapeutics. Students will learn the route that new medicines and treatments take to the clinic and the steps that ensure they are safe. Further, students will learn the role that tissue engineering plays in improving the testing of new drugs.

"We are exceedingly grateful and incredibly proud to partner with both Obatala Sciences and CellSpring to expose our future innovators to the advancements being made in biotechnology research," said STEM NOLA CEO Dr. Calvin Mackie. "This type of engagement with professionals who do this work on a daily basis is life changing. It will continue building on the interest and promise these students have already shown in STEM fields."

Dr. Mackie, a former tenured Tulane Engineering professor, founded STEM NOLA in 2014 to expose, inspire and engage communities in learning opportunities in Science, Technology, Engineering and Mathematics (STEM). The award-winning programming designs and delivers activities, programs and events. Since 2014, STEM NOLA has engaged more than 65,000 students - mostly underserved students of color - in hands-on STEM project-based learning.

"Obatala welcomes this opportunity to advance STEM NOLA's core mission and promote STEM education in our community," said Obatala Sciences Chief Scientific Officer, Dr. Jeffrey Gimble. "We are excited to have a chance to instruct and learn from the next generation of home-grown scientists recruited from our local high schools. By investing in their future, we are guaranteeing that biotech companies like ours will have a pipeline of talent to draw on in years to come."

Obatala Sciences' CEO, Dr. Trivia Frazier, agreed, saying, "We are proud to work with STEM NOLA on this project and are excited to see Dr. Mackie's continued progress building this organization."

Founded in 2017, Obatala Sciences is a biotechnology company that offers research products and services to scientists in medical industries and academia with the goal of advancing tissue engineering and regenerative medicine. Obatala makes high-quality products that researchers use to discover better therapies for diseases like obesity and diabetes that significantly impact the human population.

CellSpringdevelops blood tests to screen at-risk patients for cancer with the goal of significantly shifting the timing of a diagnosis to earlier stages. CellSpring's tests are based on a proprietary technology that effectively 'tricks' cancer cells into revealing the molecular makeup of small particles, called exosomes, that secrete into the blood. "We are proud of the work that STEM NOLA does to bring exposure, access, and opportunity to young innovators," said Dr. Christopher Millan, CellSpring CEO. "I am thrilled to have this chance to contribute to that work, and collaborating with good friends at Obatala."

Dr. Queralt Vallmaj-Martn, CELLSpring's postdoctoral researcher, added, "I'm very excited to share with NOLA high school students our passion for science and introduce them to the fascinating world of biomaterials and stem cells. Our goal is to spark inspiration in these students to discover the unknown and see that the scientific world has a great deal to offer for personal and professional development. Looking forward to our Saturdays together!"

(To interview Calvin Mackie, contact Michael K. Frisby, [emailprotected]or 202-625-4328.)

Contact:Michael K. Frisby [emailprotected] 202-625-4328

Casey Ferrand [emailprotected] (504) 810-3565

SOURCE STEM NOLA

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STEM NOLA Teams with Obatala Science & CellSpring to Expose Students to Careers and Medical Research at Biotech Startups - PRNewswire

Breakthrough gene therapies in sight | UNC-Chapel Hill – UNC Chapell Hill

Patients withgeneticlysosomal storagediseases particularly children are living longer because of better treatments. But with promising advances and longer lives comes complications, the loss of eyesight as these rare diseases take their toll over time.

Lysosomal storage diseases are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the bodys cells as a result of enzyme deficiencies, according to theNational Organization for Rare Disorders. With these diseases, patients are missing the mucopolysaccharidosis type 1 (MPS1) gene. Current therapies that allow patients to live longer dont address corneal clouding caused by these diseases. However, one Carolina-affiliated startup, RainBio, isdeveloping anovel gene therapyforMPSI corneal blindness, giving patients a chance not only for longer lives but better ones.

The companys treatment, RBIO-1, is poised to meet a large unmet medical need in ophthalmology.Co-foundedbyMatt Hirsch,associate professor of ophthalmology at the UNC Gene Therapy Center, RainBio is the only ocular gene therapy pioneer focused on blindness in the cornea. The companys adeno-associated virus vector gene therapy can directly correct cornea cellular function to maintain or restore vision in patients at risk of blindness. The companys first candidate is a groundbreaking, first-in-class lead program for corneal clouding in MPS1 patients who have vision loss.

As we look at what is being done in the ophthalmology space, we see that most [adeno-associated virus] therapeutics are focused on the back of the eye the retina, but nobody seems to be looking at the front of the eye the cornea, says Fran Martin, RainBio president. If we can provide those affected with lysosomal storage diseases a mechanism where they can see, theyd be able to have a better quality of life.

The idea for RainBios treatment started less than five years ago, when Dr. Joanne Kurtzberg, a pediatric bone marrow transplant specialist, who conducts stem cell transplants in children with lysosomal storage diseases at Duke, met with Hirsch and Samulski to figure out how to potentially address the blindness that comes with lysosomal storage disease. Kurtzberg works with stem cell therapies that can extend the longevity of life for kids affected but dont address the loss of sight. Hirsch realized if he could replace the missing gene in the cornea, those kids could regain their sight.

With our product, you inject the missing gene right into the cornea, says Martin. This allows for prevention of corneal clouding or blindness, and can even reverse the blindness or cornea clouding if the treatment is given early enough.

For kids who are losing their sight, another option is to have a cornea transplant. But those transplants dont come without risk.

Corneal transplants can sometimes result in high rejection rates and potential infection, adds Martin. We can offer an additional treatment that patients can try before moving to a transplant.

The RainBio team often collaborates with colleagues at local universities as well asJoseph Muenzer,a professor of pediatric genetics and metabolism at the School of Medicine, who treats children affected bylysosomal storage diseases.Jude Samulski, professor of pharmacology at the Gene Therapy Center in the UNC School of Medicine and a pioneer in gene therapy, also contributed to the patents for the product.

A lot of expertise in gene therapy was developed over the past few decades at UNC, says Martin. That expertise has benefited RainBio, and weve benefited from the input into the science around our product as well.

To make the best use of resources, the RainBio team benefits from collaborating withKickStart Venture Services as well as Carolinas Office of Technology Commercialization. KickStart supports faculty startup formation, business development and growth by providing coaching and mentoring, early-stage funding, connection with key service providers, management, investors and space. Both KickStart and the Office of Technology Commercialization. are part of Innovate Carolina, the University-wide initiative for innovation and entrepreneurship.

Its been a pleasure to work with KickStart and OTC to really get the guidance and resources needed as weve grown as a company, says Martin. They helped us work through the Carolina Express License and have been very supportive in helping us to understand the process as well as in providing resources as we think through our strategy as a growing company.

The Carolina Express license assists Carolina startups by streamlining and speeding the path for UNC-Chapel Hill faculty, students or staff startup founders to translate new discoveries into useful products.

Alongside Epigenos Bioscience, the RainBio team also had the opportunity in fall 2020 to present to approximately 20 advisors who gave them advice on how to continue commercializing their technology and growing their business, even in the midst of a pandemic. This is one of the programs that Kickstart adapted online to continue providing support for startups. Advice and guidance offered at advisory meetings includes anything from R&D to fundraising and strategy. Other startup companies that would like to present can get in touch with KickStart to be considered for a future meeting.

In addition, RainBio received a $100,000 loan fromNCBiotechas well as support from theMPS Society, which funded a key rabbit toxicology study. RainBio also collaborated with NC State University through co-founderBrian Gilger, who has conducted the toxicology studies at the NC State School of Veterinary Medicine.

And for patients who are ready to try RainBiosRBIO-1 treatment,Martin says the idea of a one-time dose is promising.

Although we are still testing the product, whats also unique about this treatment is that only one dose may be required. In addition, a very small amount of product around 250 times less will be needed compared to other treatments on the market for retinal blindness, she adds.

Although the company only launched in June 2020, RainBio already has pre-clinical efficacy and safety data and is poised to meet with the US Food and Drug Administration.

Even though were not even a year old, we have orphan drug designation and rare pediatric disease designation that was granted to us by the FDA, says Martin. We have pre-clinical efficacy in theMPS1canine model, and 100 percent prevention and reversal of cornea blindness.

RainBio is developing aplatform for additional treatmentsfor blinding corneal diseases.

There are several Lysosomal storage diseases that have the same cornea blindness issue, says Martin. Although its a different gene that is missing, we believe as we prove this concept inMPS1patients, then it will be translatable to otherlysosomal storagediseases.

As RainBio continues to grow and develop its product, next steps include accessing additional funding to manufacture the product and get it into the clinic.

Link:
Breakthrough gene therapies in sight | UNC-Chapel Hill - UNC Chapell Hill

FDA’s Abecma Approval a ‘Great Blessing’ for Patients with Multiple Myeloma – Curetoday.com

The Food and Drug Administrations (FDA) recent approval of the first CAR-T cell therapy, Abecma (idecabtagene vicleucel), for the treatment of multiple myeloma is extremely significant for adults who have not responded to at least four lines of therapy, according to an expert from the Dana-Farber Cancer Institute.

I think this is extremely significant (because) now its real, Dr. Nikhil Munshi, director of basic and correlative science at the Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute in Boston, said in an interview with CURE. To give an idea of when the studies were done, we knew (Abecma) worked very well, patients knew it worked very well. Now that it's commercially available, this is a real option for (patients).

In fact, Munshi noted, there has been a tremendous need for a CAR-T cell therapy in a heavily pretreated population with multiple myeloma.

In patients who have had a median of six prior lines of therapy, he highlighted, previously available therapies have induced response rates of 20% to 30%. With the highest target dose of Abecma, the overall response rate in the trial was approximately 82% and almost 40% of those patients had complete response to therapy, according to Munshi.

We are achieving deep responses as we had never hoped before, he said. And this is at a late stage. As we are beginning to now do studies that are earlier stage, we can only get better. So that's one component and the responses last.

Moreover, treatment with Abecma resulted in a progression-free survival (time from treatment initiation to disease progression, disease relapse or all-cause death) of about nine months compared to two to four months with other therapies.

I think there is a huge difference between what else is available in this space versus what we can get with (Abecma), he said. That doesn't mean what else is available is not useful, but now we have a very powerful, effective option available that we can give.

Just with any therapy, Munshi mentioned, there are some toxicities associated with the use of Abecma. The most common include cytokine release syndrome (CRS), which happens in approximately 90% of patients, and neurotoxicity. However, approximately 5% to 6% of CRS were serious or severe and around 3% of neurotoxicity were serious or severe.

Toxicities are there (and) are serious enough that we cannot take it lightly, he said. Patients would need some hospitalization most of the time for a short period, but it's very manageable.

Abecma has the potential to become a standard of care for a heavily pretreated population with multiple myeloma, according to Munshi. He likened it to cancer centers experienced in performing allogeneic stem cell transplants.

I do see that CAR-T (cell therapy) will not be restricted to 20 or 30 big centers like (Dana-Farber), where we do it enough, (but) it also will be in new course, done by reasonably medium-sized centers who can be equipped to do it, he said. Centers, which are currently doing transplant, in given time would be able to do CAR T and it would become reasonable standard of care.

Although the approval of Abecma is indicated for patients who have had at least four prior lines of therapy, there are multiple studies currently underway evaluating it in the second- and third-line setting, Munshi mentioned. Additionally, there is another study evaluating it in an upfront setting focusing on high-risk patients.

Its just a matter of time before the approvals are obtained for that phase of treatment, he said. It definitely belongs earlier on.

The approval is a great blessing for both physicians and patients, according to Munshi. He noted that Abecma is one therapy that leads to MRD negativity at a late stage and should as well at an earlier stage.

I believe this is the first step in the near future to a cure, he concluded.

For more news on cancer updates, research and education, dont forget tosubscribe to CUREs newsletters here.

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FDA's Abecma Approval a 'Great Blessing' for Patients with Multiple Myeloma - Curetoday.com

Enhanced inhibition of tumor growth using TRAIL-overexpressing adipose-derived stem cells in combination with the chemotherapeutic agent CPT-11 in…

This article was originally published here

Prostate Int. 2021 Mar;9(1):31-41. doi: 10.1016/j.prnil.2020.07.002. Epub 2020 Jul 31.

ABSTRACT

BACKGROUND: This study investigated the inhibition of tumor growth in castrate-resistant prostate cancer (CRPC)-bearing mice by tumor necrosis factor-related apoptosis-inducing ligand (TRAIL)-overexpressing adipose-derived stem cells (ADSCs) (hTERT-ADSC.sTRAIL), which was enhanced by combined treatment with CPT-11.

MATERIALS AND METHODS: An hTERT-ADSC.sTRAIL cell line was established by transfection with a lentiviral vector (CLV-Ubic) encoding the human sTRAIL gene. Quantitative polymerase chain reaction and Western blots were performed to confirm gene overexpression. An invasion study for the selective migration ability toward PC3 cells was performed. In the in vivo study, the tumor volume in mice treated with ADSC. sTRAIL and CPT-11 was measured.

RESULTS: Carboxylesterase was generated from hTERT-ADSCs. The gene expression of sTRAIL from hTERT-ADSC.sTRAIL was shown. The directional migration of ADSC.sTRAIL cells toward PC3 cells was significantly stimulated by PC3 cells in vitro (P < 0.05). In the in vitro study, the viability of PC3 cells significantly decreased in the presence of ADSC.sTRAIL (62.7 2.0%) and CPT-11 compared with that of CPT-11 alone (83.0 1.0%) at a cell ratio as low as 0.05 (PC3: ADSC.sTRAIL) (P < 0.05). The proportion of apoptotic PC3 cells significantly increased in the presence of ADSC.sTRAIL (37.2 2.1%) and CPT-11 compared with that of CPT-11 alone (16.5 1.0%) (P < 0.05). In the in vivo study, the inhibition of tumor growth in CRPC-bearing mice by TRAIL-overexpressing adipose stem cells was enhanced by combined treatment with the chemotherapeutic agent CPT-11 compared with that in the treatment with cpt-11 alone. Immunohistochemical staining of the removed tumors showed anti-TRAIL-positive cells and apoptotic bodies after hTERT-ADSC.sTRAIL treatment or combined treatment with hTERT-ADSC.sTRAIL and CPT-11.

CONCLUSIONS: Therapeutic stem cells expressing sTRAIL genes combined with CPT-11 can provide a new strategy for treating CRPC in clinical trials using the patients own ADSCs.

PMID:33912512 | PMC:PMC8053698 | DOI:10.1016/j.prnil.2020.07.002

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Enhanced inhibition of tumor growth using TRAIL-overexpressing adipose-derived stem cells in combination with the chemotherapeutic agent CPT-11 in...